Clinical Expertise Areas

Challenge

We turn patient feedback into Medicine

With approximately 7,000 rare diseases affecting 300 million people worldwide, clinical trial research in rare diseases continues to be a top priority for Genventis and many of our clients. Currently only 5% of rare diseases have an approved treatment even though the number of FDA orphan drug and biologic approvals has grown exponentially since passage of the Orphan Drug Act.

For those living with a rare disease, the impact is felt every day of a person's life. And although children make up around half of all those diagnosed, living with a rare disease requires life-long, often complex and challenging management.

Innovative and impactful solutions

Built on partnership

Our tailored approach to trial design and delivery expedites clinical development, improves patient outcomes, and ensures precision, efficiency, and safety. Let us help bring your innovative therapies to market faster and more effectively.

A dedicated division for the advancement of rare disease research and specialized clinical trial design.

In rare disease, more than any other, we rely on close collaborations and co-creation with experts from the community from start to finish. Partnerships and trust with the rare disease community is paramount to our collective success. We are working with people with rare diseases at every stage to understand what is most meaningful to them so we can design our clinical trials and medicines with this in mind

This dedication to partnership also extends beyond the patient community and into the scientific landscape. We recognise the value of external innovation and are dedicated to partnering with leading research and digital health companies to bring these to as many patients as possible.

By working together, we can build a better world for people with rare diseases.

We take the trust of the rare disease community seriously, and are working with patient organisations and people with rare diseases at every stage to understand what is most meaningful to them. That’s why 90% of our development programmes have input from patient partners – and our goal is to make that 100%.

A Sample of Genventis’s Experience in Rare Diseases

Category/Disorder

Rare Indications

Sickle Cell disease, Aplastic Anemia, Acute Myeloid Leukemia, Multiple Myeloma, Myelodysplastic syndromes, Bone Marrow Failure syndromes

Hemophagocytic Lymphohistiocytosis (HLH)

Ulcerative Colitis, Primary Sclerosing Cholangitis

Blood Disorders including some blood cancers

Immune System/Immune Deficiency

Autoimmune

Lysosomal Storage Disorders

Neurology

Other Rare diseases/disorders

Fabry disease, Mucopolysaccharidosis II (MPS II or Hunter Syndrome), Niemann-Pick Type C, Cystinosis

Bronchopulmonary Dysplasia (BPD), Limb-Girdle Muscular Dystrophies, Apnea of Prematurity